Weekly use of the experimental, long-acting therapy efanesoctocog alfa safely and effectively prevents bleeds in adolescents and adults with severe hemophilia A, according to top-line, one-year data from the Phase 3 XTEND-1 trial. The therapy — being jointly developed by Sanofi and Sobi — was also superior to approved preventive therapies…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…

An experimental therapy may help remove harmful antibodies that reduce the effectiveness of factor replacement therapies in hemophilia A, potentially offering a new way to overcome one of the most significant complications of standard care. The therapy uses chimeric autoantibody receptor T cells, or CAAR-T cells. These are…

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…

uniQure N.V. has updated the clinical data from its ongoing clinical trial of a gene therapy for the treatment of patients with hemophilia B. The results indicate sustained improvement by all patients at a low-dose, with durable levels of  Factor IX (FIX) gene activity for several weeks after treatment. The…

Prophylactic replacement therapy to prevent bleeds was more cost-effective than on-demand treatment of active bleeds for children and adolescents in China with moderate or severe hemophilia A without inhibitors, a study reported. The superior cost-effectiveness of prophylaxis over on-demand treatment was mainly driven by the on-demand costs of treating…

Hemophilia B patients who switched to Rebinyn (nonacog beta pegol), an extended half-life factor IX (FIX) replacement therapy, had similar or better responses than with their previous standard or extended half-life FIX replacement therapies, a real-world study in Canada suggested. The study, “Switching to nonacog beta…

Gene therapy company uniQure released new data showing that its lead therapy AMT-060 for severe and moderately severe hemophilia B is safe and effective for up to two years, with adults requiring fewer doses of replacement therapy and showing a marked decrease in the spontaneous bleeding rate. The results support uniQure’s clinical…

Switching to Rebinyn (nonacog beta pegol) from other standard or extended half-life replacement therapies led to “robust” reductions in annual treatment costs among hemophilia B patients in Canada, a study based on real-world data reported. Its findings add to an earlier and real-world analysis of patients who…